Should terminally ill patients have the right to try experimental treatments?

The emotional, knee-jerk response most of us have to that question is: “Of course, what have they got to lose?”

But who should be able to access drugs, and when, is actually one of the thorniest ethical issues and hottest political issues in health care today.

In the past year, 17 U.S. states have passed “right-to-try” laws, with little political opposition and even less critical examination.

These laws, championed by the libertarian Goldwater Institute, are based on the premise that “terminal patients shouldn’t have to ask the government for permission to save their own lives,” in the words of the group’s president, Darcy Olsen.

The assumption here is that only the patient has anything to lose (or gain). That’s not true. Neither is the argument that the paperwork for gaining access to experimental drugs now is too onerous.

The U.S. Food and Drug Administration has a program, called Expanded Access to Investigational Drugs for Treatment Use, which grants about 1,000 requests a year. (This initiative started in the early days of AIDS, as shown in the movie Dallas Buyers Club, which actually spurred the new right-to-try movement.)

Health Canada has a similar program, Special Access Programme – Drugs; last year there were 15,994 requests of which 87 per cent were granted. (However, the SAP includes all drugs not yet approved in Canada, not just those in experimental trials.)

Ultimately, however, it is the drug company that decides if drugs are released early – meaning before they are approved by regulators.

There are a few reasons for reticence. Drugs are tested in three phases: In Phase I, basic safety and dosage are examined; Phases II and III focus on actual effectiveness and side effects.

Of 100 new drugs that go into Phase I trials, only about 20 move to Phase III. That’s because 50 per cent do not work, 20 per cent are outright harmful and 30 per cent do not proceed for strategic reasons (such as not working better than current drugs, or having side effects that would make them hard to market).

U.S. laws give patients the right to demand any drug that has passed Phase I trials – and that information is easy to find in the Internet age. But what the statistics tell us is that most people who will get experimental drugs will not be helped, and a good number will actually be harmed.

Meanwhile, providing experimental drugs on demand can undermine clinical trials (in which the drug is tested against a placebo to see if it actually works) and delay drugs that do work getting to market, where they will be accessible to all patients.

Allowing the sidestepping of the regulatory process also opens the door to predatory charlatans who will exploit the dying with impunity. (Most right-to-try laws absolve practitioners of liability if they treat a patient with an investigational drug.)

What tends to happen these days is that desperate patients and their families glom onto a promising study and launch social-media campaigns to demand access to a drug.

One of the most oft-cited cases is that of seven-year-old Joshua Hardy, who suffered a life-threatening infection after a bone-marrow transplant. His family wanted access to the experimental drug brincidofovir, but the company, Chimerix, initially refused, saying that supplies were limited and it could undermine its clinical trial.

Both traditional and social media picked up on the story of the “evil” pharma company denying a boy life and it was bombarded with responses, up to and including death threats to the chief executive officer. Ultimately, Chimerix created a whole new trial to focus on Josh’s type of illness; he was included and got better.

But ethicists pointed out that providing special access to those who have the means and savvy to berate companies is also unfair.

So, last week, the health-care behemoth Johnson & Johnson tried a new tack, appointing an independent committee, led by world-renowned ethicist Arthur Caplan, to review requests for early access to medications, in the hope of making it fairer and more consistent.

It remains to be seen if that will assuage the impatient and those who believe the hopes of an individual trump all other concerns.

People who are terminally ill deserve compassion, but in our bid to be compassionate, we should not be offering up false hope, nor undermining the benefits to others.

As much as we would like to, we cannot legislate magical cures, nor legislate away suffering.

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